Genetic Diseases and Gene Therapy - Professor Mark …：遗传性疾病和基因疗法教授马克….pptVIP

Gene Therapy Disappointments In 1999 a boy died due to an immune response to an adenovirus gene therapy vector. Four children have developed cancer due to a retrovirus gene therapy vector My Research: Building a better Vector Adenovirus shell delivers genes HSV-like virus protein tethers DNA to chromosome to keep genes in cell without integration Your Goals: Learn more about gene therapy by completing a becoming a SPACE DOCTOR! Gene Therapy Vector Engineering Project. First groups to complete the Space Doctor activity gets to chose from the genetic diseases we talked about today. Use what you have learned and the Vector Toolbox to choose the best vector for treating the disease. Tomorrow Learn about the techniques used to genetically engineer gene therapy vectors. Gene Therapy Vector Engineering Project. Use paper models to simulate the process of engineering your gene therapy vector. * Contemporary Issue – Genetic Disorders and Gene Therapy Lesson Goals To understand the nature of genetic diseases. To differentiate between chromosomal and single gene diseases. To understand the nature of Down Syndrome and diagnostic methods to detect it. To understand the important points of heredity including dominant and recessive alleles. To understand how to predict heredity using a Punnett square. To understand the nature and heritability of Huntington Disease, Cystic Fibrosis, and Sickle Cell Disease. To understand the nature of genetic testing and it’s future application to diagnose diseases and the probability of getting genetic diseases. To understand the nature of gene therapy and it’s future application to cure diseases. Key Terms; chromosome chromosomal disease meiosis nondisjunction Down syndrome karyotype amniocentesis autosomal disease gene allele dominant recessive co-dominant widow’s peak genotype phenotype Huntington disease cystic fibrosis sickle cell disease * Advanc